2025 LeukoAware Community Partner Spotlight

Welcome to our 2025 LeukoAware Community Partner Spotlight Series! We are shining a light on the incredible organizations supporting families and individuals affected by leukodystrophy. Partnerships are essential in the rare disease space, and we’re excited to highlight our partners’ missions, impact, and dedication for Leukodystrophy Awareness Month.

CureCADASIL
CureCADASIL is an organization making a difference every day for those impacted by leukodystrophy. Their dedication is evident not just in what they do, but in how they describe their work themselves.
In their own words, CureCADASIL’s mission is simple, but powerful: we are a patient-led organization committed to advancing CADASIL (cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy) research by uniting patients, researchers, and clinicians in the search for a cure. Our organization holds tight to the age-old adage that knowledge is power, but we go further by recognizing that raising awareness is equally vital. Through collaboration, we aim to identify key areas of research that will deepen our understanding of CADASIL, guiding strategic partnerships that accelerate research, increase awareness, and maximize our impact on efforts to find treatments that slow disease progression or ultimately cure the disease.
The following quote is from Sheila Connor, on behalf of her husband, Tom:
“He [Tom] had his first two strokes in 2015, and then for a year and a half everything was quiet. Then, he started having strokes every three or four months. In 2022, at the beginning of the year, he had a pretty significant stroke and hospice was called in. He passed away in July of 2022. Since then, I’ve joined the Community Advisory Group through CureCADASIL, and we’re working with Dr. Elahi and the Chan Zuckerberg Patient Initiative [to] raise awareness.”
Visit https://curecadasil.org to learn more!
Global DARE Foundation
The Global DARE Foundation is a champion for the leukodystrophy community dedicated to improving the lives of those impacted by Adult Refsum Disease (ARD) around the world. Their work is driven by a clear and powerful purpose.
The Global DARE Foundation’s mission is to promote worldwide awareness and a better quality of life for all who are diagnosed with Adult Refsum Disease. Their goal is to support research, education initiatives, awareness campaigns, and advocacy efforts that create meaningful change.
Susan Kuranoff, Vice President, Global DARE Foundation shares:
“At the Global DARE Foundation, we know that awareness is the first step toward change. By shining a light on Adult Refsum Disease and other leukodystrophies, we not only help families find answers sooner, but also inspire research, improve care, and connect people who might otherwise feel alone. When the world understands these rare conditions, we open the door to earlier intervention, better treatments, and the hope our community deserves.”
And the impact of their work is reflected in the voices of the community they serve. As one community member shares:
“The Global DARE Foundation has provided a way for patients and their families to feel a connection to, and to share experiences with, other patients, their families and friends.” – Richard P.
To learn more about the Global DARE Foundation visit: defeatadultrefsumeverywhere.org
 
 
EndAxD
End AxD is an organization dedicated to the Alexander Disease community. Their fight against this ultra-rare leukodystrophy is powered by passion, persistence, and purpose.
From Jennifer Pearson, President, End AxD:
“End AxD is fiercely committed to serving the Alexander Disease community—accelerating research toward treatments and a cure, advocating for and empowering patients and families. As an ultra-rare leukodystrophy, Alexander Disease often leaves families navigating a painful diagnostic odyssey. Raising awareness isn’t just important, it’s essential. It helps to unite fragmented efforts under one umbrella, strengthens the patient registry, fuels research momentum, and offers hope where it’s needed most. United, we are unstoppable. Together we will End AxD.”
From their community:
“When my son Asa was first diagnosed, I was desperate for information to better explain what was going on. Google search was pretty grim, but the shining light for me was finding End AxD. Reading the patient testimonials and seeing pictures of kids like my son gave me hope for his future. The info was easy to understand. I actually printed several things off to show our family when we were breaking the news that our son has this disease. We appreciate all the work y’all are doing to help get info out to share awareness and all the work being done to aid in finding treatments for our loved ones.” – Lesley F.
Learn more about End AxD at: www.endaxd.org
National Tay-Sachs & Allied Diseases Assoication (NTSAD)
National Tay-Sachs & Allied Diseases Association, Inc. (NTSAD) is an organization dedicated to supporting families and driving progress for the leukodystrophy community. Their mission and impact extend beyond research; they are a lifeline for families navigating rare diseases.
In their own words:
National Tay-Sachs & Allied Diseases Association (NTSAD) leads the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases by driving research, forging collaboration, and fostering community. Supporting families is at the center of everything we do.
NTSAD supports families through personalized services, including 1:1 emotional support, educational resources, and guidance throughout diagnosis and care. They bring families together at their Annual Family Conference and work tirelessly to raise awareness, foster collaboration, and fund research that improves quality of life and advances the search for effective treatments.
From the board:
“NTSAD’s mission to find treatments and eventually a cure for Tay-Sachs, Canavan, GM1, and Sandhoff diseases is deeply personal to me as the father of Cameron, age 10, who has Canavan Disease. NTSAD was a lifeline for my family following our son’s diagnosis; resources and community it offers to patients and their families is invaluable. Increasing public awareness for the leukodystrophy community is crucial to the support, funding, and research efforts of patient advocacy organizations like NTSAD, with the added benefit of empowering patients and their families by fostering understanding and reducing the sense of isolation that all too often can accompany these devastating diseases.”
—Jon L., NTSAD Board Vice President, Dad to Cameron
From the community:
“When we found NTSAD, everything changed. We had just left Chile to give our son with Canavan disease the best life possible, but we felt lost in a new country, language, and reality. NTSAD welcomed us with open arms and gave us not only guidance and support, but a true family we can always count on.”
—Francisca C., mom to Arturo
Visit www.ntsad.org to learn more!
Alex, The Leukodystrophy Charity
(Alex TLC)
Alex TLC (Alex, The Leukodystrophy Charity), lifting voices, supporting research and raising awareness for the leukodystrophy community.
In Their Own Words:
Our support focuses on the similarities of these conditions, embracing all affected within a community of Tender Loving Care. We support research into these rare conditions and raise awareness through campaigns targeting the general public and the medical profession. By working in collaboration with leukodystrophy specialists and other rare disease charities, we aim to improve best practices within healthcare systems.
“ I have lived experience of the fear, isolation and confusion a leukodystrophy diagnosis can bring, after my mum was diagnosed in 2021. Alex TLC are there for those living with leukodystrophy and their families to provide support and information, when this is usually lacking from local services. Just having someone there who understands what you are going through, who can guide the way is vital when facing something that initially feels so lonely and hopeless.
Raising awareness is crucial, as many people are misdiagnosed with more common neurological diseases, and due to the genetic nature of the majority of leukodystrophies, it is important that families know exactly what is affecting their loved one(s) as more family members may go on to develop symptoms, or be carriers of the gene. Having a name for their condition also gives people access to appropriate treatments or emerging medical trials, something which would not be possible if they were left in the dark.
Through our work with the medical community, we are hopeful that in the future people won’t face delays in diagnosis or misdiagnosis, and be offered appropriate care in a timely manner, reducing the stress and trauma that can arise from a lengthy diagnostic journey that is currently still common. Through our collaboration with research initiatives like LEUKOLABS, we look forward to a day when effective treatments are available and a leukodystrophy diagnosis doesn’t devastate lives to the extent it does today.” – Fiona Willis, Alex TLC Team Member – Fundraising, Communications and Design Officer
“As a Trustee for Alex TLC and someone with lived experience of leukodystrophy, I know first hand when a family first hears the word ‘leukodystrophy’ they are often met with a lack of answers. This can be incredibly isolating and scary – not just for the individual but the whole family. That’s why Alex TLC’s mission is so important. We’re not just about research or improving patient care, we’re about creating a community of support and hope. Raising awareness isn’t just a goal, it’s a lifeline. It means earlier diagnoses, better access to treatments and ensuring no family has to face this journey alone”. – Emma Hadfield-Hudson, Alex TLC Trustee
“I can’t thank you [our Director of Support] and Jenna [our Counsellor] enough for the support you have provided to me and my family since my diagnosis. I really don’t know what we (especially me) would have done without you both for the past year. It’s amazing to know that you are both there for me in the run up to my return to work following substantial sickness. Thanks so much.” – patient community member
“We were put in touch with Karen [our Director of Support] when our lives were flipped upside down and at an all-time low and she quickly became someone we go to for any help with doctors and consultants or even just a catch-up but this is always met the same way. She helps us by coming to appointments to make sure our Multidisciplinary Team meetings go to plan and recently getting hold of consultants for us. She is invaluable and we see her as a part of our journey. We love sharing our highs and lows. She’s the voice of caring when you need it and a beacon of knowledge when you want it.” – patient community member
Learn more about Alex TLC at:  www.alextlc.org 
Cure MSD Foundation
United MSD Foundation Cure MSD , building hope for those living with Multiple Sulfatase Deficiency (MSD). Their purpose is as bold as it is urgent, and they express it best themselves.
In their own words: 
Our mission is to cure Multiple Sulfatase Deficiency. The United MSD Foundation is more than an organization—it is a global community of families, researchers, care providers, and advocates working together to:
✔️ Ensure patients have a seat at the table in policy and access discussions
✔️ Increase research funding for rare diseases
✔️ Accelerate the development and approval of treatments
✔️ Center ethical considerations in decision-making by policymakers and industry partners.
Our work spans research, family support, and advocacy, including:
• Partnering on gene therapy projects, cellular models, natural history studies, and biomarker development
• Advancing newborn screening for lysosomal storage disorders, including MSD—critical for early intervention, clinical trial enrollment, and ongoing family support until therapies are available
• Connecting families with resources, expert care, and one another, ensuring no family faces MSD alone
The Foundation’s Executive Director, Sarah Cortell Vandersypen, reflected on their role in the larger leukodystrophy movement: “Every family we support is a vital part of the larger story of leukodystrophy. By connecting MSD families worldwide with expert care, research opportunities, and one another, we are breaking down barriers for those living with multiple sulfatase deficiency and shining a light on the broader challenges and urgent needs faced by all leukodystrophy communities. Awareness fuels understanding, and understanding drives the research, resources, and hope our children deserve.”
At the 2024 Biennial International MSD Scientific and Family Conference, MSD mom Natalie Sheppard shared the impact of this work and the United MSD Foundation’s supportive community:
“The conference has been amazing, actually being around other parents who get it… [Families in the MSD community] all know how hard it is for each other, and there’s a lot of shared pain and shared grief. And that’s really hard to sit with, but it’s just harder to sit alone.” “One of the first things they tell you when you get your diagnosis is, ‘Oh, this is super rare,’ but then they handed me this whole booklet, introduced me to the community, the Facebook page. I really appreciated that they had these resources ready for me at the diagnosis meeting. … I was surprised to find that there was this community that was really active and that clinical trials are on the horizon. That’s amazing. What a time to be diagnosed, when there’s this much hope, thanks to organizations like the United MSD Foundation… as opposed to what it must have been like before [organizations like this existed].”
Visit www.unitedmsd.org to learn more.
CTX Alliance
 CTX Alliance, with a mission of providing education, support, advocacy, and promoting research for Cerebrotendinous Xanthomatosis (CTX) patients, their families, and medical professionals who treat and stuQdy this rare disease. “The CTX Alliance has been a great help, allowing me to collaborate with others, connect with physicians who specialize in my needs, access a community of resources, and find comfort in knowing I am not alone, with a whole community willing to support me in any way possible—that’s what it’s all about.” – CTX Alliance Community Member “Leukodystrophy affects families in profound ways, and raising awareness is essential. Greater understanding leads to earlier diagnosis, which allows for timely therapeutic interventions and better quality of life. Our mission ensures that patients and families know they are not alone in this journey.” – CTX Alliance Board Member To learn more, check out their website at https://ctxalliance.org/
Cure LBSL

Cure LBSL’s mission is to advance awareness of LBSL, support patients and families and ultimately cure this leukodystrophy that slowly robs most people of their ability to walk. The nonprofit funds LBSL research around the world, natural history studies and potential gene therapies and drug repurposing. “LBSL is an ultra-rare disease with an ultra-long name: leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation. Our patients — including my own daughter — are some of the most resilient people I’ve ever met. They’re the reason why we’re working so hard to turn a cure from a dream into a reality, and we’re closer than ever to unlocking a gene therapy. I can’t wait for the day where we get to announce that we cured LBSL — and that day is coming.” — Morgan Voigt, Cure LBSL’s executive director In their own words: “A Cure for Ellie (now Cure LBSL) was the first source of help we found after our family received the LBSL diagnosis. Ellie and her family gave us the strength and hope we desperately needed when everyone else was telling us there was none.” — Stephanie in America, mom of two boys with LBSL “We dream that a cure will be found as soon as possible.” — Irina in Ukraine, mom of three children with LBSL “To any and everyone trying to help figure this out, thank you. It means more than you will ever know.” — Josh in America, who was diagnosed with LBSL at age 9 Learn more at https://www.curelbsl.org

PMD Foundation

This family-driven foundation proactively serves those affected by Pelizaeus-Merzbacher Disease (the PMD community) by supporting programs of education, research, service, and advocacy. We are dedicated to providing patients and their families with information about their disease and assistance in identifying sources of medical care, social services, and genetic counseling; establishing a communications network among families; increasing public awareness and acting as an information source for healthcare providers; and promoting research into causes, treatment, prevention, and cure of PMD. From the Board Chair, Storm Greenway: “As a father of a child with PMD I found myself looking for anything to help my son. Once I learned about the disease I felt compelled to truly do something to help him, that is what drew me to the PMD Foundation initially. Although it is an ambitious task, the strides that have been made in PMD research are making an idea that once seemed impossible now be just within reach. It is rewarding to both have hope for my son and be in a position to help others with PMD. I’ve also found a unique bond shared with the other families who know what I am going through. I’m so thankful that we’ve been able to create a community for our warriors and a platform for families to share their story.” From the Vice Chair, Angi Dunham: “Awareness is the first step toward change. With awareness comes research, resources, and the chance to give families the answers and hope they deserve.” To learn more about the PMD Foundation visit: https://www.pmdfoundation.org/

ELA International

ELA International: A European organization dedicated to financing leukodystrophy research and supporting families across multiple countries. In Their Own Words: ELA International focuses on two core missions:
✔ Financing research on leukodystrophies
✔ Supporting families through our European chapters (Belgium, France, Germany, Italy, Luxembourg, Spain, and Switzerland) From the Scientific Community: “ELA should be extremely proud of the fact that they have built the field of leukodystrophy research. Without ELA, we wouldn’t be having many of the discussions around outcomes biomarkers trials, and even the basic pathophysiology of some rare diseases. ELA helped fund some of these early studies. It is a critical force in the field, something to be very proud of.” —Florian Eichler, Director of the Leukodystrophy Service at Massachusetts General Hospital, Professor of Neurology at Harvard Medical School, President of the ELA Scientific Committee From the Board: “Should we add days to life, or life to days? People living with leukodystrophy deserve both. That is why ELA will continue tirelessly to support medical research and affected families.” —Guy Alba, Chairman of ELA International Board Learn more at: https://elainternational.eu/en/

Disclaimer: The community partners listed on this page represent those collaborating with the ULF specifically for the 2025 LeukoAware campaign.
This is not a comprehensive list of all organizational partners.

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